Suggested remit: To appraise the clinical and cost effectiveness of vutrisiran within its marketing authorisation for treating transthyretin amyloidosis with cardiomyopathy
 
Status In progress
Technology type Medicine
Decision Selected
Reason for decision Anticipate the topic will be of importance to patients, carers, professionals, commissioners and the health of the public to ensure clinical benefit is realised, inequalities in use addressed, and help them make the best use of NHS resources
Process STA Standard
ID number 6470

Provisional Schedule

Committee meeting 08 July 2025
Expected publication 12 November 2025

Project Team

Project lead Leena Issa

Email enquiries

External Assessment Group Centre for Reviews and Dissemination and Centre for Health Economics, University of York

Stakeholders

Companies sponsors Alnylam Pharmaceuticals (vutrisiran)
Others Department of Health and Social Care
  NHS England
Patient carer groups Amyloidosis UK
  Arrythmia Alliance
  Atrial Fibrillation Association
  British Liver Trust
  Cardiomyopathy UK
  Cardiovascular Care Partnership
  Circulation Foundation
  Gene People
  Genetic Alliance UK
  HEART UK
  Liver4Life
  Pumping Marvellous
  Somerville Foundation
  South Asian Health Foundation
  Specialised Healthcare Alliance
Professional groups Association of Genetic Nurses and Counsellors
  British Cardiovascular Society
  British Geriatrics Society
  British Nuclear Cardiology Society
  British Society of Echocardiography
  British Society for Gene and Cell Therapy
  British Society for Genetic Medicine
  Haemochromatosis UK
  National Heart and Lung Institute
  Primary Care Cardiovascular Society
  Royal College of General Practitioners
  Royal College of Nursing
  Royal College of Pathologists
  Royal College of Physicians
  Royal Pharmaceutical Society
  Royal Society of Medicine
  UK Clinical Pharmacy Association
  Vascular Society of Great Britain and Ireland
Associated public health groups Public Health Wales
  UK Health Security Agency
Comparator companies Pfizer (tafamidis)
General commentators All Wales Inherited Metabolic Disease Service
  All Wales Therapeutics and Toxicology Centre
  Allied Health Professionals Federation
  Association of Renal Industries
  Board of Community Health Councils in Wales
  British National Formulary
  Care Quality Commission
  Cell and Gene Therapy Catapult
  Department of Health - Northern Ireland
  Healthcare Improvement Scotland
  Medicines and Healthcare products Regulatory Agency
  National Association of Primary Care
  National Pharmacy Association
  National Services Division
  NHS Confederation
  Scottish Medicines Consortium
  Welsh Government
  Welsh Health Specialised Services Committee
Relevant research groups Cochrane Heart Group
  Genomics England
  MRC Clinical Trials Unit
  National Centre for Cardiovascular Preventions and Outcomes
  National Institute for Health Research

Timeline

Key events during the development of the guidance:

Date Update
12 December 2024 Referral
25 November 2024 Invitation to participate
04 October 2024 - 01 November 2024 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 6470
04 October 2024 In progress. Scoping commencing
15 July 2024 Awaiting development. Status change linked to Topic Selection Decision being set to Selected
15 July 2024 Topic selection

For further information on our processes and methods, please see our CHTE processes and methods manual